Illinois-based AveXis is a clinical-stage gene therapy company, developing novel treatments for patients suffering from rare neurological genetic diseases. One-time penny stock player Vaxart made market waves Tuesday morning when the company announced it had dosed the first patients in the Phase I study for its oral Covid-19 vaccine candidate. The company is currently focused on finding new small molecule binding sites — which may affect protein degradation, inhibition or both — to drug the undruggable. Last October, the FDA OK’d a semi-controversial new antibiotic from Shionogi. The company issued 4,300,000 shares at $19.00-$21.00 per share. The grant includes $2.62 million for Polyphor’s “hit-to-lead” stage and $15.82 million in milestones. The group already had 50 translational scientists at work and a GMP facility. We are optimistic on this deal, following the success of gene-therapy company BGNE last week. In one of two large studies for the drug, known chemically as cefiderocol and commercially as Fetroja, more patients died in the treatment arm than the control arm. In a statement put out Monday evening, the pharma giant said that it had paused further dosing, noting that “the participant’s illness is being reviewed and evaluated by the ENSEMBLE independent Data Safety Monitoring Board as well as our internal clinical and safety physicians.”. The idea was to tap into a line of AAV9 vector gene therapies that were being developed at UT Southwestern, particularly from the labs of Steven Gray and Berge Minassian. Gene therapy trials, of course, can cost a fair bit of cash. That round — which also added deep-pocket player ARCH to the list of backers — came up with $77 million for the next step in the long journey toward the clinic, a nice add to the A round that launched the company.
A few weeks ago Kite and Allogene founder Arie Belldegrun jumped into the expanded syndicate for a Boston-based biotech called Dewpoint Therapeutics — a Polaris-birthed venture that’s styled itself as a drug development pioneer out to craft a major pipeline. “But we’ve forgotten that phosphotases are also important.”. Now we hear that Dewpoint has recruited Ameet Nathwani to the executive suite as the new CEO, who’s taking the helm from Polaris managing partner Amir Nashat, who brought the company into existence. Investors have showed a willingness to put upwards of $200 million to companies that have yet to bring a drug into the clinic. It recently bagged a second award of up to $18.44 million for work on what it calls a new class of antibiotics to fight drug-resistant Gram-negative bacteria. UPDATED: Another safety incident triggers a halt to a pivotal Covid-19 vaccine study as J&J slams on the brakes, UPDATED: Vaxart was served a grand jury subpoena in July over its controversial role in OWS-funded preclinical study, Novartis bet $6B on the idea — now Versant, venBio have $45M to birth a platform play for radiopharmaceuticals, News briefing: Novo eyes the next kinases in a Finnish startup; Boehringer Ingelheim brings a bispecific to the clinic, Months after achieving unicorn status, Orca steers into the fast lane with an RMAT designation for cell therapy candidate, Pair of Lancet studies give final word on a promising Shionogi antibiotic that turned out to be 'as good' as the other 'suboptimal' options, Polyphor bags second grant in two years for a new class of antibiotics to fight drug-resistant bacteria, Third Rock-backed Cedilla reels in $57.6M for small molecule programs to fight cancer. (AVXS) raised $86 million in an IPO on Thursday, February 11th 2016. The early stage, European VC firm announced yesterday they led a $9 million seed round for Rappta Therapeutics, a Finnish biotech developing inhibitors for an enzyme called PP2A — protein phosphatase 2 — that Bakker thinks now hold the same kind of drug targeting potential as kinases did two decades ago for cancer. They licensed 15 gene therapies in epilepsy, neurodevelopment and neurodegenerative disorders, with an option to license 4 more. Biotechs, including those relatively fresh off launch, have been going public at a frenzy since the pandemic began.
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